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CCX168

Phase 2

Vasculitis | Small molecule | Immunology |Amgen Inc.|Last Updated: Mar 13, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment115
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01363388A Study to Evaluate the Safety and Efficacy of CCX168 in Subjects With ANCA-Associated VasculitisPHASE2 COMPLETED 67Sep 1, 2011Jan 1, 2016Mar 13, 202544 Austria, Belgium +8
NCT05984251A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of CCX168 in Healthy ParticipantsPHASE1 COMPLETED 48Dec 21, 2009Apr 11, 2011Aug 9, 20231 Switzerland
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Study Endpoints
Primary Endpoints
Proportion of Subjects Achieving Disease Response at Day 85
Baseline to Day 85

Disease response is defined as BVAS percentage reduction from baseline of at least 50% plus no worsening in any body system component.

Number of Participants Experiencing Adverse Events (AEs)
Up to 43 days
Number of Participants Experiencing Clinically Significant Changes in Laboratory Parameters
Up to 29 days
Number of Participants Experiencing Clinically Significant Changes in Electrocardiogram (ECG) Parameters
Up to 29 days
Number of Participants Experiencing Clinically Significant Changes in Vital Sign Parameters
Up to 29 days
Secondary Endpoints
Proportion of Patients Achieving Renal Response at Day 85
Baseline to Day 85
Proportion of Subjects Achieving Disease Remission at Day 85
Day 85
Percent Change From Baseline to Day 85 in BVAS
Baseline to Day 85
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
PlaceboPLACEBO_COMPARATORPlacebo plus a full dose of oral glucocorticoids for steps 1 and 2 of the study
CCX168EXPERIMENTAL30 mg Active study medication, plus either two-thirds reduced dose of oral glucocorticoids for step 1 of the study, or no oral glucocorticoids for step 2 of the study
Cohort 1EXPERIMENTALDuring Period 1, participants will receive a single dose of CCX168 1 mg or placebo. During the second study period, participants will receive the same dose as during the first period but once daily (QD) for a period of 7 days continuously.
Cohort 2EXPERIMENTALDuring Period 1, participants will receive a single dose of CCX168 3 mg or placebo. During the second study period, participants will receive the same dose as during the first period but QD for a period of 7 days continuously.
Cohort 3EXPERIMENTALDuring Period 1, participants will receive a single dose of CCX168 10 mg or placebo. During the second study period, participants will receive the same dose as during the first period but QD for a period of 7 days continuously.
Cohort 4EXPERIMENTALDuring Period 1, participants will receive a single dose of CCX168 30 mg or placebo. During the second study period, participants will receive the same dose as during the first period or placebo twice daily (BID) for a period of 7 days continuously.
Cohort 5EXPERIMENTALDuring Period 1, participants will receive a single dose of CCX168 100 mg or placebo. During the second study period, participants will receive the same dose as during the first period or placebo BID for a period of 7 days continuously.
Interventions
NameTypeDescription
PlaceboDRUGBID for 84 days
CCX168DRUGBID for 84 days
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites44

Key Inclusion Criteria: * Clinical diagnosis of granulomatosis with polyangiitis (Wegener's), microscopic polyangiitis or renal limited vasculitis * Male and postmenopausal or surgically sterile female subjects aged at least 18 years with new or relapsed AAV where treatment with cyclophosphamide or...

Countries:AustriaBelgiumCzechiaFranceGermanyHungaryNetherlandsPolandSwedenUnited KingdomSwitzerland
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